It's Time We Get Serious About Faster Drug Approval

The Food and Drug Administration is the necessary evil that's there to keep the checks and balances in place for the drug innovation process -- but the regulatory agency is damned if it does and damned if it doesn't. When Merck's (NYSE: MRK) arthritis medication Vioxx was recalled in 2004 and linked to more than 27,000 cardiovascular events, the FDA was criticized for not seeing the drug's serious early warning signs. On the other hand, the simple fact that it takes an average of 12 years from conceptualization of a drug in a laboratory to eventual approval speaks of the ridiculously difficult process involved to get a drug approved. Like I said, damned if it does and damned if it doesn't.

We can do better "Why are you bringing this up," you may be wondering? This one's a bit personal to me as I've personally witnessed the intangible costs that disease has exacted on family and friends.

I lost my mother to non-small-cell lung cancer in August 2010, long before Celgene's (Nasdaq: CELG) Abraxane added NSCLC as an additional indication. Today, I'm writing this article from the hospital bedside of my good friend Scott Cruz who's been battling brain cancer for 23 years, since he was just 3 years old. Roche's (NASDAQOTH: RHHBY) Avastin was recently combined with his current treatment, Temodar, manufactured by Merck, to produce a positive progression-free survival benefit in phase 3 trials over just Temodar alone. However, this potential combination comes too little too late for Scott because of the rigorous FDA approval process -- even for already-approved drugs that are targeting additional indications.

I realize that I'm not the only person who's lost a loved one to a disease that seems to have discoveries emerging at a snail's pace, and I surely won't be the last, but the pace and pathways by which the FDA is requiring biotech companies to go from innovation to marketing is simply inadequate. We need to get serious about faster drug approval, and it needs to happen now.

Baby steps We've seen some discernible steps to speed up the approval process for innovation to marketing with the passing of the Food and Drug Administration Safety and Innovation Act, or FDASIA. This bill, signed into law in July, will allow the FDA to collect user fees from brand-name drug makers, generic drug makers like Mylan (Nasdaq: MYL) -- the biggest generic producer which had previously been exempt from any user fees -- and medical device companies, to conduct and expedite safety and efficacy reviews of crucial pipeline drugs and devices.

One specific provision of the FDASIA was the TREAT Act, introduced by Sen. Kay Hagan. The specifics of the TREAT Act are that unmet and serious medical diseases will now face an accelerated review process in the attempt to get these potentially life-saving treatments to patients earlier.

These are great steps, and I applaud the Senate, House of Representatives, and president for putting these provisions into law in order to get treatments to those who truly need it. But, just as this is a step in the right direction, it's also an arduously slow process that hasn't gone nearly far enough.

This can't be as good as it getsTo begin with, I'd like to point to my colleague Brian Orelli who hit one of my key gripes right on the head back in late September. We've seen a select few expedited approvals -- most notably Medivation's (Nasdaq: MDVN) prostate cancer drug Xtandi, which was approved months ahead of its PDUFA date -- but where are the expedited non-approvals?

It's understandable to assume that not every drug is going to meet the FDA's rigorous safety and efficacy criteria, but why can't we get this information back to the formulating companies earlier? Just like the theory of "If the black box can survive a plane crash, then why don't we make the entire plane out of the same material," I think to myself, if fast-track status gives a company a more intimate hand-in-hand approach to drug development, why can't we fast-track all drugs, use these user fees to expand the workforce of the FDA, and get biotech companies working side-by-side with FDA regulators to get drugs to market quicker? We could possibly eliminate half of all drug safety and manufacturing hold-ups, if not more!

Another factor that astounds me is that we've seen genome sequencing costs go from north of $10,000 just a decade ago to just $1,000. With the ability to accurately and rapidly turn genome data around in a day, why aren't we seeing biotechs, big pharmaceutical companies, and the FDA focusing on using this technology to move studies out of the lab even quicker than they are now? Realistically, we're losing three to four years on lab work and pre-clinical studies before they even make it to clinical trials. We have the technology to drastically shorten this time period, and regulators are effectively twiddling their thumbs.

There also has to be a better way of sharing information across the aisles -- and I'm not talking about Democrats and Republicans in this case. Specifically, I can't understand why we can't get the European Medicines Agency -- or EMA -- the FDA of Europe, and the FDA to cooperate to bring drugs to market quicker. I fully understand that the two agencies have varying goals, and approval in Europe is usually seen as easier than in the United States, but it's incredibly irritating to see drugs get held up for years in the U.S. approval process when they're already approved in Europe. These two agencies need to find a way to put their knowledge together and work as a team.

I'm open for change in many different respects, and these suggestions above are merely a combination of Brian's idea and my own ideas. What I can tell you is this: Having spent many months in and out of hospitals over the past three years, people like my mother and Scott have received great care, but they deserve and can/could have received better. The FDA and lawmakers are finally starting to take steps in the right direction, but we're only at the beginning of a major crossroads in the transformation of the drug approval process.

What suggestions do you have to expedite the FDA drug approval process? Post your thoughts, no matter how grandiose, in the comments section below.

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Fool contributor Sean Williams has no material interest in any companies mentioned in this article. He dedicates this article to his friend, Scott Joseph Cruz. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.

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The sorry fact is that many many lawsuits happen if an approved new drug does not work, or causes other complications that were not expected.

Those individuals, like your mom and friend, should have these life saving drugs available as a last resort. If the drug companies are protected from future lawsutes due to immature usage prior to Govt. approval, maybe these could be available on fast track for those in dire need. It could be a win win for both parties. Of course it could be a losing situation for the patient.

Just like the papers you sign before an operation you need to hold harmless to the provider of the drugs that have not yet been approved.

This article appears to blame one and the only one source for delay in drug development and drug approvals.

The following are just a few points to counter the FDA blame

Based on the total number of active Phase II and III clinical trials in the US/FDA jurisdiction, Tufts study estimates that the pharmaceutical industry spends between $4 billion and $6 billion each year on procedures that generate extraneous clinical trial data. Meaning unnecessary work!!!

According to Dr. Langer (NY times dated Nov24th)and his quote “Very often when you are going for real innovation,” “you have to go against prevailing wisdom, and it’s hard to go against prevailing wisdom when there are people who have been there for a long time and you have some vice president who says, ‘No, that doesn’t make sense.’ ” Referring to Big Pharma!! Another quote about Big Pharma "While drug companies employ huge research and development teams, they may not be as freewheeling and nimble"

Basically, lot of waste in spending huge amounts of money on pet projects, even though the earlier signals during the drug development indicated lack of safety and efficacy e.g. Torcetrapib from Pfizer should have been killed at earlier stage of phase II instead of killing at late phase III stage-800 millions spent. Also, some of the true hard working development staff who warned of these signals were fired or transferred to other projects.

The other main reason is not listening to the warnings from the FDA during the many interactions during the early development from IND to NDA (which are not public knowledge due to confidentiality), which sometimes leads to additional trials for discarding the earlier FDA and other regulatory Agency recommendations. Please don't blame the FDA, blame the big pharma and the arrogant management in general for having a lackadaisical approach to the research and development, appointing and promoting incompetent highly paid ‘yes sir’ individuals, or introducing politics in R&D, and no respect for the genuine, hard working and analytical minded development staff including savvy regulatory professionals, who get thrown out for alerting the early warning signals!!!